Fabry Disease Clinical Trial
Official title:
A Randomized, Open-label, Active Comparator, 2-arm, Prospective Study to Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
Primary Objective: To assess reduction of plasma lyso-GL3 level after switch to agalsidase beta from agalsidase alfa Secondary Objectives: - To assess reduction of kidney podocyte GL3 content after switch to agalsidase beta from agalsidase alfa - To assess reduction of GL3 content in endothelial skin cells after switch to agalsidase beta from agalsidase alfa - To assess change in renal function after switch to agalsidase beta from agalsidase alfa - To assess disease severity and clinical changes after switch to agalsidase beta from agalsidase alfa - To assess improvement in symptoms of Fabry disease after switch to agalsidase beta from agalsidase alfa
The study will have a screening period of up to 9 weeks. Eligible participants will be randomized to switch to agalsidase beta or to continue agalsidase alfa in a 1:1 ratio for a period of 12 months (52 weeks). ;
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